shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Title:

shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Document Type and Number:

WIPO Patent Application WO/2023/172535

Kind Code:

A3

Abstract:

Provided herein are compositions and methods for activating expression from the paternally-inherited allele of UBE3A in Angelman syndrome using viral vector delivery of short hairpin RNAs. Provided herein are compositions and methods for reducing or eliminating expression of SNORD115 and UBE3A-ATS in Angelman syndrome using viral vector delivery of short hairpin RNAs.

Inventors:

CHAMBERLAIN STORMY (US)
GERMAIN NOELLE (US)
PERRINO PETER (US)

Application Number:

PCT/US2023/014680

Publication Date:

November 30, 2023

Filing Date:

March 07, 2023

Export Citation:

Click for automatic bibliography generation Help

Assignee:

UNIV CONNECTICUT (US)
OVID THERAPEUTICS INC (US)

International Classes:

C12N15/13; A61K31/7105; A61P25/00; C12N15/864; C12N15/867

Domestic Patent References:

WO2020061528A1	2020-03-26
WO2013098381A1	2013-07-04

Foreign References:

US20210332368A1	2021-10-28
US11198869B2	2021-12-14
US20200291485A1	2020-09-17

Other References:

DATABASE NUCLEOTIDE ANONYMOUS : "Eukaryotic synthetic construct chromosome 15", XP093115473, retrieved from NCBI

Attorney, Agent or Firm:

STEEN, Jeffrey S. (US)

Download PDF:

View/Download PDF PDF Help

Previous Patent: shRNA TARGETING UBE3A-ATS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Next Patent: OPTICAL TRANSCEIVER WITH INTEGRATED DISPERSION COMPENSATION FOR HIGH BIT RATE APPLICATIONS